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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05667493




Registration number
NCT05667493
Ethics application status
Date submitted
12/07/2022
Date registered
28/12/2022

Titles & IDs
Public title
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Scientific title
An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Secondary ID [1] 0 0
2022-000826-74
Secondary ID [2] 0 0
ION-682884-CS12
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Eplontersen

Experimental: Eplontersen - Eplontersen will be administered once every month by sub-cutaneous (SC) injection for up to 36 months or 6 months after eplontersen is approved and available in the site's country, whichever occurs first.


Treatment: Drugs: Eplontersen
Eplontersen will be administered by SC injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
Timepoint [1] 0 0
Baseline up to 36 months
Primary outcome [2] 0 0
Change From Baseline in Platelet Count
Timepoint [2] 0 0
Baseline up to 36 months
Primary outcome [3] 0 0
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
Timepoint [3] 0 0
Baseline up to 36 months
Primary outcome [4] 0 0
Change From Baseline in Urine Protein Creatinine Ratio (UPCR)
Timepoint [4] 0 0
Baseline up to 36 months
Primary outcome [5] 0 0
Change From Baseline in AST
Timepoint [5] 0 0
Baseline up to 36 months
Primary outcome [6] 0 0
Change From Baseline in ALT
Timepoint [6] 0 0
Baseline up to 36 months
Primary outcome [7] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters
Timepoint [7] 0 0
Baseline up to 36 months
Primary outcome [8] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Thyroid-Stimulating Hormone (TSH)
Timepoint [8] 0 0
Baseline up to 36 months
Primary outcome [9] 0 0
Percentage of Participants With Anti-Drug Antibodies (ADA)
Timepoint [9] 0 0
Baseline up to 36 months
Secondary outcome [1] 0 0
Change From Baseline in Transthyretin (TTR) Serum Levels
Timepoint [1] 0 0
Baseline up to 36 months
Secondary outcome [2] 0 0
Change From Baseline in 6-minute Walk Test (6MWT)
Timepoint [2] 0 0
Baseline up to 36 months
Secondary outcome [3] 0 0
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)
Timepoint [3] 0 0
Baseline up to 36 months
Secondary outcome [4] 0 0
Change From Baseline in Short Form Health Survey Questionnaire (SF-36)
Timepoint [4] 0 0
Baseline up to 36 months
Secondary outcome [5] 0 0
Change From Baseline in 5 Level EuroQol - 5 Dimension (EQ-5D-5L)
Timepoint [5] 0 0
Baseline up to 36 months
Secondary outcome [6] 0 0
Change From Baseline in Biomarker: N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)
Timepoint [6] 0 0
Baseline up to 36 months
Secondary outcome [7] 0 0
Change From Baseline in Biomarker: High Sensitivity Cardiac Troponin T (hs-cTnT)
Timepoint [7] 0 0
Baseline up to 36 months

Eligibility
Key inclusion criteria
1. Satisfactory completion of Treatment Period and the End of Treatment Visit of the Index Study (ION-682884-CS2) OR diagnosis of ATTR-CM and satisfactory participation on ISIS 420915- CS101 study as judged by the Investigator and Sponsor.
2. Investigator is willing to treat the participant with open-label eplontersen.
3. Willingness to adhere to vitamin A supplementation per protocol.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Permanently discontinued study drug administration while participating in the Index Study (ION 682884-CS2) or IST (ISIS 420915-CS101 Study).
2. Have any new condition or worsening of an existing condition that in the opinion of the Investigator or Sponsor would make the participant unsuitable for enrolment, or which could interfere with the participant participating in or completing the study, including the need for treatment with medications disallowed in the Index Study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/11/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2029
Actual
Sample size
Target
1400
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
West AustraliaWA
Recruitment hospital [1] 0 0
Advara HeartCare Murdoch - Murdoch
Recruitment hospital [2] 0 0
Advara HeartCare - Joondalup
Recruitment hospital [3] 0 0
Box Hill Hospital - Clayton
Recruitment hospital [4] 0 0
Royal Hobart Hospital - Hobart
Recruitment hospital [5] 0 0
Advara HeartCare - Joondalup - Joondalup
Recruitment hospital [6] 0 0
Liverpool Hospital - Liverpool
Recruitment hospital [7] 0 0
Westmead Hospital - Westmead
Recruitment hospital [8] 0 0
Princess Alexandra Hospital - Woolloongabba
Recruitment postcode(s) [1] 0 0
6150 - Murdoch
Recruitment postcode(s) [2] 0 0
6027 - Joondalup
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment postcode(s) [4] 0 0
7000 - Hobart
Recruitment postcode(s) [5] 0 0
2170 - Liverpool
Recruitment postcode(s) [6] 0 0
2145 - Westmead
Recruitment postcode(s) [7] 0 0
4102 - Woolloongabba
Recruitment outside Australia
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United States of America
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Arizona
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California
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District of Columbia
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Georgia
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Illinois
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Indiana
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Kansas
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Maryland
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Massachusetts
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Ciudad Autónoma De Buenos Aires
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Graz
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Athens
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Haifa
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Israel
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Rehovot
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Firenze
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Pavia
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Pisa
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Italy
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Roma
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Barcelona
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Majadahonda
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Murcia
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Spain
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Santiago De Compostela
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Göteborg
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Sweden
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Skellefteå
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Hampstead
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United Kingdom
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London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ionis Pharmaceuticals, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
AstraZeneca
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Ionis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
(844) 520-3239
Fax 0 0
Email 0 0
ionisATTRCM2study@clinicaltrialmedia.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05667493